Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT01960244 |
| Other study ID # |
Pro00045568 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
October 2013 |
| Est. completion date |
December 2025 |
Study information
| Verified date |
November 2023 |
| Source |
The Familial Hypercholesterolemia Foundation |
| Contact |
MARY CARD, MBA |
| Phone |
6265834674 |
| Email |
mcc[@]thefhfoundation.org |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational [Patient Registry]
|
Clinical Trial Summary
The CASCADE-FH Registry is a national, multi-center initiative that will track the therapy,
clinical outcomes, and patient-reported outcomes over time. The registry represents a
collaboration between The Familial Hypercholesterolemia Foundation, the Duke Clinical
Research Institute, lipid specialists, cardiologists, primary care providers, quality
improvement personnel, and patients, all aiming to increase FH awareness, promote optimal
disease management, and improve FH outcomes.
Description:
The FH Foundation CASCADE-FH Registry will implement an enrollment framework which is
characterized by three possible points of contact: 1) Clinic enrollment 2) Self-enrollment
through an online patient portal, and 3) Electronic Health Record (EHR) identification.
Pathway 1: Clinic-Based Screening and Enrollment During the initial study phase, a number of
specialized lipid clinics across the US will participate in the CASCADE-FH registry. FH
patients at these sites who meet inclusion will enroll. Each site will be required to receive
IRB approval and obtain patient consent. Once the initial set of specialized lipid clinics
has demonstrated acceptable feasibility for patient enrollment and engagement, additional
sites will be recruited into the registry.
Data collection For data entered at clinical sites, the primary source of information will be
the patient's medical record. Baseline data will elements to be abstracted and entered.
Data elements entered by self-enrolled patients in the online patient portal will include a
subset of clinical information as well as questions on quality of life, disease-related
anxiety, and depression. A short survey to assess patient understanding of FH health risks,
available treatment options, and family member screening will also be included. The patient
questionnaire was designed to be free of clinical jargon and pilot-tested by FH patient
volunteers to ensure ease of use by participants.
Followup data collection For patients enrolled at clinical sites, providers will be asked to
update information at yearly intervals. Medical records will be reviewed to assess changes in
medications, occurrence of major adverse cardiovascular events, hospitalizations, genetic
testing, and laboratory values updated since the last date of data entry. Follow-up data will
be collected yearly for 3 years following initial enrollment. Self-enrolled patients may
update data at any time by accessing the patient portal. Updated information on current
medication regimens, clinical events, and quality of life will be collected. However,
reminder emails will be sent to all self-enrolled patients on an annual basis to ensure
uniform entry of follow up information.
Longitudinal Outcomes Serial lipid values will be a key outcome of interest and will be
examined to assess the adequacy of lipid modifying therapies to achieve target LDL values.
Longitudinal outcomes of interest will include medication changes, occurrence of major
adverse cardiovascular events, and mortality. Primary patient-reported outcomes of interest
will include notification and screening of family members, treatment satisfaction,
disease-related perceptions, and quality of life measurements.
Statistical Considerations The CASCADE-FH Registry will collect clinician-reported
information to characterize treatment patterns and outcomes among FH patients. Because this
study is not-hypothesis driven and no specific medical therapies or treatment interventions
are being compared, formal prospective calculations of sample sizes are not necessary.
However, we will periodically assess variations in lipid management, clinical events, and
patient-reported outcomes to evaluate temporal changes in these variables. Standard
statistical approaches commonly used in observational analyses will be utilized.
Data Feedback and Quality Improvement Sites participating in the CASCADE-FH Registry will
receive annual data feedback reports that will highlight treatment patterns, serial lipid
values, and clinical outcomes for their enrolled patients compared with the national results.
These reports will be designed to facilitate quality improvement interventions at
participating sites designed to improve the treatment and outcomes of FH patients.
Self-enrolled patients will have the opportunity to download their reported data directly as
well as through pre-programmed self-feedback, electronic reports that can be accessed at any
time.
